New targeted pill shows promise for rare blood cancer
NCT ID NCT03580655
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 2 study tested avapritinib, a daily tablet that targets a specific genetic mutation, in 107 people with advanced systemic mastocytosis (a rare blood cancer). The main goal was to see how many patients had their tumors shrink or symptoms improve. Results suggest the drug may help control the disease and ease symptoms, but more research is needed.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
avapritinib (a targeted cancer drug taken as a tablet)
What this could lead to
If successful, this could provide a new treatment option that shrinks tumors and improves symptoms for people with advanced systemic mastocytosis.
What could go wrong
This is a single-arm Phase 2 study with no comparison group, so results may not hold up in larger trials. The drug may cause side effects and not work for everyone.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
Salerno, 84131, Italy
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Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative
Florence, 50134, Italy
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Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde
Glasgow, G12 0YN, United Kingdom
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CHU Toulouse - Hôpital Larrey
Toulouse, 31059, France
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Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona
Verona, 37134, Italy
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
London, SE1 9RT, United Kingdom
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Herbert Irving Comprehensive Cancer Center
New York, New York, 10032, United States
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Huntsman Cancer Institute
Salt Lake City, Utah, 84112, United States
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München
Munich, 81675, Germany
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Mays Cancer Center
San Antonio, Texas, 78229, United States
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Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie
Vienna, 1090, Austria
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Odense University Hospital, Department of Haematology
Odense, DK-5000, Denmark
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Oslo University Hospital-Rikshospitalet, Hematology
Oslo, 0372, Norway
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Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
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Stanford Cancer Institute
Stanford, California, 94305, United States
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The University of Kansas Cancer Center
Westwood, Kansas, 66205, United States
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Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation
Aachen, 52074, Germany
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University Medical Center Groningen (UMCG)
Groningen, 9713 GZ, Netherlands
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Pennsylvania, Abramson Cancer Center
Philadelphia, Pennsylvania, 19104, United States
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University of Texas, MD Anderson Cancer Center
Houston, Texas, 77030, United States
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Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie
Hamburg, 20246, Germany
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Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie
Leipzig, 04103, Germany
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Universitätsmedizin Mannheim III. Medizinische Klinik
Mannheim, 68167, Germany
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Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku
Wroclaw, 50-367, Poland
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Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii
Gdansk, 80-214, Poland
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
Toledo, 45071, Spain